Notizia - Recent Pharma, Healthcare and Biotech Happenings
Jun 24, 2025
Table of Contents
Incyte has announced that the FDA has approved MONJUVI (tafasitamab-cxix) in combination with rituximab and lenalidomide for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL). This makes it the first FDA-approved CD19- and CD20-targeted immunotherapy combination for this patient population. The approval is based on the pivotal Phase III inMIND trial, which showed a significant improvement in progression-free survival (PFS) with the MONJUVI regimen compared to the control arm. Patients receiving the combination therapy had a median PFS of 22.4 months versus 13.9 months in the control arm, marking a hazard ratio of 0.43 (P<0.0001).
The safety profile of the MONJUVI combination was evaluated in 546 patients. Serious adverse reactions occurred in 33% of patients, with infections being the most common. Grade 3 or 4 laboratory abnormalities included decreased neutrophils and lymphocytes. The approval marks the second U.S. indication for MONJUVI, which is also approved for relapsed or refractory diffuse large B-cell lymphoma (DLBCL) in combination with lenalidomide.
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“Patients living with relapsed or refractory FL have been waiting for new options that improve progression-free survival without substantial increase in side effects. Based on the data from the inMIND trial, today’s approval brings the first CD19- and CD20-targeted immunotherapy combination and a potential new treatment standard,” said Hervé Hoppenot, CEO of Incyte. “This second U.S. approval for MONJUVI reinforces our commitment to advancing innovation for the lymphoma community.”
“The FDA approval of MONJUVI in combination with rituximab and lenalidomide marks a significant advancement, offering a chemotherapy-free option that has demonstrated a meaningful reduction in the risk of disease progression,” said Dr. Christina Poh, Assistant Professor of Medicine at the University of Washington and Fred Hutchinson Cancer Center.
Follicular lymphoma, the second most common type of non-Hodgkin lymphoma, accounts for up to 30% of NHL cases. While typically slow-growing, it presents a therapeutic challenge due to frequent relapses and the risk of progression to aggressive forms of lymphoma. “We are pleased that the FDA has approved tafasitamab, part of a treatment combination offering a new option for patients living with this chronic disease,” said Dr. Mitchell Smith, Chief Medical Officer, Follicular Lymphoma Foundation.
Gilead Sciences has received FDA approval for YEZTUGO (lenacapavir), a long-acting, injectable HIV-1 capsid inhibitor for pre-exposure prophylaxis (PrEP) to reduce the risk of sexually acquired HIV in adults and adolescents weighing at least 35kg. YEZTUGO becomes the first and only PrEP option in the U.S. that requires just two injections per year. The approval is based on Phase III PURPOSE 1 and PURPOSE 2 trials, where ≥99.9% of participants remained HIV negative throughout the study.
Despite the availability of PrEP for over a decade, data from the CDC shows that only 36% of eligible individuals were prescribed PrEP as of 2022. Uptake remains particularly low among women, Black and Latino communities, and people living in the U.S. South, due to barriers such as stigma, adherence issues, and limited awareness. With over 100 new HIV diagnoses each day in the U.S., the approval of a more convenient, long-acting prevention method marks a major advancement in addressing these gaps.
“This is a historic day in the decades-long fight against HIV. YEZTUGO is one of the most important scientific breakthroughs of our time… it could transform HIV prevention,” said Daniel O’Day, Chairman and CEO, Gilead Sciences. “With FDA approval and collaboration with our partners, we’re now closer than ever to ending the HIV epidemic.”
“YEZTUGO could be the transformative PrEP option we’ve been waiting for—offering the potential to boost PrEP uptake and persistence,” said Dr. Carlos del Rio, Emory University. “A twice-yearly injection addresses key barriers like adherence and stigma, especially for those on daily oral PrEP regimens.”
Incyte announced that the FDA has extended the review period for its sNDA of ruxolitinib cream (Opzelura®) for the treatment of children aged 2–11 with mild to moderate atopic dermatitis (AD). The PDUFA date is now set for September 19, 2025, following the submission of additional chemistry, manufacturing, and controls (CMC) data related to the 0.75% formulation in response to an FDA request.
The sNDA is supported by positive data from the Phase III TRuE-AD3 trial, which met its primary endpoint. A higher percentage of patients using Opzelura achieved Investigator’s Global Assessment-treatment success (IGA-TS) compared to vehicle. A key secondary endpoint—EASI75 at Week 8—was also successfully met. The safety profile remained consistent with no serious infections, cardiovascular events, malignancies, or thromboses reported during the 8-week vehicle-controlled period.
p“Atopic dermatitis is a chronic, immune-mediated skin condition that can be difficult to manage, particularly in young children,” said Dr. Steven Stein, Chief Medical Officer at Incyte. “We are confident in Opzelura’s potential as a non-steroidal topical treatment for pediatric AD and are working closely with the FDA to support its review.”
The most common treatment-related adverse event observed was mild application site pain (2.7%), which did not lead to discontinuation. If approved, Opzelura could offer a steroid-free treatment alternative for pediatric patients, a significant step forward in addressing unmet needs in younger populations affected by eczema.
Archeus Technologies has announced that the FDA has cleared its Investigational New Drug (IND) application for ART-101, a novel radiopharmaceutical therapy developed for the imaging and treatment of metastatic castration-resistant prostate cancer (mCRPC). This approval will allow Archeus to initiate a Phase I clinical trial later in 2025. ART-101 is a PSMA-targeting small molecule with higher tumor uptake and lower off-target accumulation compared to currently approved agents, potentially offering improved efficacy and reduced toxicity.
Developed in collaboration with the University of Wisconsin–Madison and the Wisconsin Alumni Research Foundation, ART-101 is part of Archeus’ growing pipeline of theranostic radiopharmaceuticals. The therapy is designed to deliver alpha particle-emitting isotopes more safely and effectively than existing options, offering a differentiated mechanism for treating PSMA-positive prostate cancer.
“This IND clearance marks an important milestone for Archeus as we prepare to initiate the first of multiple clinical trials set to begin this year,” said Evan Sengbusch, Ph.D., CEO of Archeus Technologies. “ART-101 represents a promising approach to targeting PSMA-positive prostate cancer with the potential to deliver meaningful clinical benefit and reduced side effects for patients.”
In addition to ART-101, Archeus received IND clearance for ARC-706 and its companion diagnostic ARC-166 in October 2024. The company plans to advance both assets as a theranostic pair, especially for use alongside immunotherapies in resistant cancer types. Preclinical results suggest the duo may elicit curative responses and even immune memory in cancers typically unresponsive to immunotherapy.
Cycle Pharmaceuticals announced that the FDA has approved HARLIKU (nitisinone) tablets for the treatment of alkaptonuria (AKU), an ultra-rare genetic metabolic disorder. HARLIKU, launching in July 2025, becomes the first and only FDA-approved therapy to reduce urine homogentisic acid (HGA) levels in adults with AKU. This approval marks a major milestone for patients suffering from complications like early-onset osteoarthritis, ochronosis, and organ damage, significantly impacting mobility and quality of life.
The approval is based on data from a randomized, no-treatment controlled study of 40 AKU patients conducted by the National Human Genome Research Institute (NHGRI) at the NIH. The study, led by Dr. Wendy J. Introne, MD, demonstrated that long-term treatment with nitisinone significantly improved pain, energy levels, and physical function over three years, as measured by the 36-Item Short-Form Survey.
“We are deeply grateful for Cycle’s collaboration with Dr. Wendy Introne, Dr. Bill Gahl, and the broader team at the NIH,” said Steve Fuller, Chief Strategy Officer of Cycle Pharmaceuticals. “We look forward to making HARLIKU available to U.S. AKU patients as soon as possible and remain committed to supporting the AKU community to the fullest extent of our capabilities.”
Dr. Introne added, “The approval of HARLIKU is an important advance for the AKU community. Our scientific team has translated decades of research into launching nitisinone as a new treatment option, and we stand hopeful that it can ease the significant burden of AKU.”
With this milestone, HARLIKU becomes Cycle Pharmaceuticals’ eighth commercial product in the U.S., reinforcing the company’s focus on advancing therapies for rare and underserved diseases.
Article in PDF
Jun 24, 2025
Table of Contents
Incyte has announced that the FDA has approved MONJUVI (tafasitamab-cxix) in combination with rituximab and lenalidomide for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL). This makes it the first FDA-approved CD19- and CD20-targeted immunotherapy combination for this patient population. The approval is based on the pivotal Phase III inMIND trial, which showed a significant improvement in progression-free survival (PFS) with the MONJUVI regimen compared to the control arm. Patients receiving the combination therapy had a median PFS of 22.4 months versus 13.9 months in the control arm, marking a hazard ratio of 0.43 (P<0.0001).
The safety profile of the MONJUVI combination was evaluated in 546 patients. Serious adverse reactions occurred in 33% of patients, with infections being the most common. Grade 3 or 4 laboratory abnormalities included decreased neutrophils and lymphocytes. The approval marks the second U.S. indication for MONJUVI, which is also approved for relapsed or refractory diffuse large B-cell lymphoma (DLBCL) in combination with lenalidomide.
“Patients living with relapsed or refractory FL have been waiting for new options that improve progression-free survival without substantial increase in side effects. Based on the data from the inMIND trial, today’s approval brings the first CD19- and CD20-targeted immunotherapy combination and a potential new treatment standard,” said Hervé Hoppenot, CEO of Incyte. “This second U.S. approval for MONJUVI reinforces our commitment to advancing innovation for the lymphoma community.”
“The FDA approval of MONJUVI in combination with rituximab and lenalidomide marks a significant advancement, offering a chemotherapy-free option that has demonstrated a meaningful reduction in the risk of disease progression,” said Dr. Christina Poh, Assistant Professor of Medicine at the University of Washington and Fred Hutchinson Cancer Center.
Follicular lymphoma, the second most common type of non-Hodgkin lymphoma, accounts for up to 30% of NHL cases. While typically slow-growing, it presents a therapeutic challenge due to frequent relapses and the risk of progression to aggressive forms of lymphoma. “We are pleased that the FDA has approved tafasitamab, part of a treatment combination offering a new option for patients living with this chronic disease,” said Dr. Mitchell Smith, Chief Medical Officer, Follicular Lymphoma Foundation.
Gilead Sciences has received FDA approval for YEZTUGO (lenacapavir), a long-acting, injectable HIV-1 capsid inhibitor for pre-exposure prophylaxis (PrEP) to reduce the risk of sexually acquired HIV in adults and adolescents weighing at least 35kg. YEZTUGO becomes the first and only PrEP option in the U.S. that requires just two injections per year. The approval is based on Phase III PURPOSE 1 and PURPOSE 2 trials, where ≥99.9% of participants remained HIV negative throughout the study.
Despite the availability of PrEP for over a decade, data from the CDC shows that only 36% of eligible individuals were prescribed PrEP as of 2022. Uptake remains particularly low among women, Black and Latino communities, and people living in the U.S. South, due to barriers such as stigma, adherence issues, and limited awareness. With over 100 new HIV diagnoses each day in the U.S., the approval of a more convenient, long-acting prevention method marks a major advancement in addressing these gaps.
“This is a historic day in the decades-long fight against HIV. YEZTUGO is one of the most important scientific breakthroughs of our time… it could transform HIV prevention,” said Daniel O’Day, Chairman and CEO, Gilead Sciences. “With FDA approval and collaboration with our partners, we’re now closer than ever to ending the HIV epidemic.”
“YEZTUGO could be the transformative PrEP option we’ve been waiting for—offering the potential to boost PrEP uptake and persistence,” said Dr. Carlos del Rio, Emory University. “A twice-yearly injection addresses key barriers like adherence and stigma, especially for those on daily oral PrEP regimens.”
Incyte announced that the FDA has extended the review period for its sNDA of ruxolitinib cream (Opzelura®) for the treatment of children aged 2–11 with mild to moderate atopic dermatitis (AD). The PDUFA date is now set for September 19, 2025, following the submission of additional chemistry, manufacturing, and controls (CMC) data related to the 0.75% formulation in response to an FDA request.
The sNDA is supported by positive data from the Phase III TRuE-AD3 trial, which met its primary endpoint. A higher percentage of patients using Opzelura achieved Investigator’s Global Assessment-treatment success (IGA-TS) compared to vehicle. A key secondary endpoint—EASI75 at Week 8—was also successfully met. The safety profile remained consistent with no serious infections, cardiovascular events, malignancies, or thromboses reported during the 8-week vehicle-controlled period.
p“Atopic dermatitis is a chronic, immune-mediated skin condition that can be difficult to manage, particularly in young children,” said Dr. Steven Stein, Chief Medical Officer at Incyte. “We are confident in Opzelura’s potential as a non-steroidal topical treatment for pediatric AD and are working closely with the FDA to support its review.”
The most common treatment-related adverse event observed was mild application site pain (2.7%), which did not lead to discontinuation. If approved, Opzelura could offer a steroid-free treatment alternative for pediatric patients, a significant step forward in addressing unmet needs in younger populations affected by eczema.
Archeus Technologies has announced that the FDA has cleared its Investigational New Drug (IND) application for ART-101, a novel radiopharmaceutical therapy developed for the imaging and treatment of metastatic castration-resistant prostate cancer (mCRPC). This approval will allow Archeus to initiate a Phase I clinical trial later in 2025. ART-101 is a PSMA-targeting small molecule with higher tumor uptake and lower off-target accumulation compared to currently approved agents, potentially offering improved efficacy and reduced toxicity.
Developed in collaboration with the University of Wisconsin–Madison and the Wisconsin Alumni Research Foundation, ART-101 is part of Archeus’ growing pipeline of theranostic radiopharmaceuticals. The therapy is designed to deliver alpha particle-emitting isotopes more safely and effectively than existing options, offering a differentiated mechanism for treating PSMA-positive prostate cancer.
“This IND clearance marks an important milestone for Archeus as we prepare to initiate the first of multiple clinical trials set to begin this year,” said Evan Sengbusch, Ph.D., CEO of Archeus Technologies. “ART-101 represents a promising approach to targeting PSMA-positive prostate cancer with the potential to deliver meaningful clinical benefit and reduced side effects for patients.”
In addition to ART-101, Archeus received IND clearance for ARC-706 and its companion diagnostic ARC-166 in October 2024. The company plans to advance both assets as a theranostic pair, especially for use alongside immunotherapies in resistant cancer types. Preclinical results suggest the duo may elicit curative responses and even immune memory in cancers typically unresponsive to immunotherapy.
Cycle Pharmaceuticals announced that the FDA has approved HARLIKU (nitisinone) tablets for the treatment of alkaptonuria (AKU), an ultra-rare genetic metabolic disorder. HARLIKU, launching in July 2025, becomes the first and only FDA-approved therapy to reduce urine homogentisic acid (HGA) levels in adults with AKU. This approval marks a major milestone for patients suffering from complications like early-onset osteoarthritis, ochronosis, and organ damage, significantly impacting mobility and quality of life.
The approval is based on data from a randomized, no-treatment controlled study of 40 AKU patients conducted by the National Human Genome Research Institute (NHGRI) at the NIH. The study, led by Dr. Wendy J. Introne, MD, demonstrated that long-term treatment with nitisinone significantly improved pain, energy levels, and physical function over three years, as measured by the 36-Item Short-Form Survey.
“We are deeply grateful for Cycle’s collaboration with Dr. Wendy Introne, Dr. Bill Gahl, and the broader team at the NIH,” said Steve Fuller, Chief Strategy Officer of Cycle Pharmaceuticals. “We look forward to making HARLIKU available to U.S. AKU patients as soon as possible and remain committed to supporting the AKU community to the fullest extent of our capabilities.”
Dr. Introne added, “The approval of HARLIKU is an important advance for the AKU community. Our scientific team has translated decades of research into launching nitisinone as a new treatment option, and we stand hopeful that it can ease the significant burden of AKU.”
With this milestone, HARLIKU becomes Cycle Pharmaceuticals’ eighth commercial product in the U.S., reinforcing the company’s focus on advancing therapies for rare and underserved diseases.
